A radical new gene therapy for Parkinson’s disease has shown promise in tests on patients – but evidence that it works is still missing, researchers report today.
The therapy seeks to convert healthy brain cells to produce the brain chemical dopamine, the shortage of which is responsible for many of the symptoms of the debilitating disease.
Some 15 patients took part in the French study of the gene therapy, known as ProSavin. The treatment uses an inert virus and three dopamine-making genes.
The virus was injected into the striatum region of the brain where movement is controlled.
The results are reported in The Lancet today.
In the study, three different doses were tested on the 15 patients.
The patients experienced no more than moderate side-effects such as "on-off" experiences, the doctors say. At periods of six months and a year after treatment, patients were assessed at periods of at least 12 hours after their last use of dopamine medication. This suggested "significant improvements" in movement abilities, researchers said.
The researchers, led by Professor Stéphane Palfi, from AP-HP, Groupe Henri-Mondor Albert-Chenevier in Créteil, France, say the findings show "promise" – but similar effects have been found in other trials for placebo treatments.
They say the treatment "might provide an effective long-term treatment without the onset of behavioural complications."
But, writing in the journal, Jon Stoessl from the University of British Columbia, Vancouver, Canada, says the treatment may not deal with the real challenges of Parkinson’s disease.
He says: "Despite numerous imperfections, we have reasonably good treatments for the motor manifestations of Parkinson’s disease in the form of levodopa, infusion therapies, and deep brain stimulation.
"The challenge of Parkinson’s disease is the management of non-motor problems, many of which have a non-dopamine basis. The therapy described here will not address these issues."
The Lancet 10 January 2014 [abstract]
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